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Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): A multicentre, open-label, non-randomised phase 3 study

The Lancet Haematology Apr 23, 2019

Pipe SW, et al. - In this phase 3, multicenter, open-label, two-stage study performed with adults and adolescents with hemophilia A, researchers evaluated emicizumab prophylaxis given as one dose every 4 weeks, irrespective of factor VIII (FVIII) inhibitor status. Study participants were patients 12 years and old undergoing treatment with either FVIII concentrates or bypassing agents for severe congenital hemophilia A (<1% of normal FVIII activity in blood) or hemophilia A with FVIII inhibitors. In the run-in cohort, patients recruited from three sites in Japan and Spain were included, and a subsequent expansion cohort comprised patients recruited from 17 sites in Australia, Belgium, Japan, Poland, Spain, and the US. Good tolerability, as well as a clinically meaningful bleed control, were associated with emicizumab given once every 4 weeks. For people with hemophilia A, better adherence to effective prophylaxis and a lower treatment burden could result from this regimen, improving patient care and ultimately having the potential to reduce the development of secondary complications.

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